This grant will support Drs. Mendell and Rodino-Klapac and their team's work in exploring gene therapy as a potential treatment for Duchenne. Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, also announced in January that it has entered a research agreement with Nationwide Children’s Hospital on their microdystrophin gene therapy program.

The initial trial, expected to go into Phase 1/2a trial in late 2017, will be conducted at Nationwide Children’s. PPMD has committed the $2.2 million grant to the trial, with support from additional Duchenne foundations and families. Sarepta will also provide support to the trial through a separate research agreement with Nationwide Children’s, and will have an exclusive option to license the program. PPMD's grant provided incentive for Sarepta to help expand and accelerate this opportunity.